Overview

• The Phase 1/2 study followed 29 participants for up to 36 months, with the high-dose cohort showing an estimated 75% reduction in clinical decline versus external controls.
• The therapy, AMT-130, is delivered via a 12–18 hour neurosurgical procedure that injects a viral vector into the striatum under real-time MRI guidance.
• Neurofilament light in cerebrospinal fluid, a marker of neuronal injury, fell by an average of about 8% after postoperative spikes, supporting a biological effect.
• Most adverse events were related to the operation and resolved, according to investigators, though the invasive delivery and perioperative biomarker surges remain concerns.
• Data are preliminary and not peer-reviewed, with small numbers and reliance on historical comparators; uniQure is recruiting larger multicenter trials and plans an FDA submission in 2026, which, if successful, could lead to the first genetic therapy for Huntington’s disease.